FDA Grants Orphan Drug Status to TGV-Inhalonix’s Inhaled Solution as a Possible Treatment for Resistant CF Lung Infections

TGV-Inhalonix announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to its antimicrobial agent as a possible treatment for antibiotic-resistant bacterial lung infections in patients with cystic fibrosis (CF).

Mul-1867 is a nebulized solution designed for easy self-administration. According to the company, it is the first inhaled antimicrobial being developed against multi-drug–resistant bacteria and fungi that can be the cause of serious lung infections in CF patients.

“This is a breakthrough for our novel drug candidate, which has shown good results in early studies,” Dr. Victor Tetz, chief scientific advisor at TGV-Inhalonix, said in a recent press release.

“Previous studies have shown that Mul-1867 holds promise as a treatment for life-threatening pulmonary infections from Pseudomonas aeruginosa, Burkholderia cepacia, and other bacteria,” said Dr. George Tetz, drug development advisor at TGV-Inhalonix. “Our research revealed that Mul-1867 is more effective against antibiotic-resistant pathogens as compared with other drugs commonly used for the treatment of infections in cystic fibrosis patients.”

The Orphan Drug Act provides a special status to a drug or biological product to treat a rare disease or condition, defined as one affecting fewer than 200,000 people in the U.S. at any given time. Orphan Drug designation qualifies the drug’s sponsor for various development incentives, including tax credits for qualified clinical testing.

Mul-1867 was designated an orphan drug upon review of experimental data provided the FDA by TGV-Inhalonix. The company, in the release, says that Mul-1867’s  mechanism of action has a broad potential to treat fungal and bacterial infections, including respiratory tract infections in patients with fungal pneumonia, chronic obstructive pulmonary disease (COPD), and ventilator-associated pneumonia (VAP). Data assessing its efficacy have been published in several scientific journals, and were presented at a number of microbiology and infectious disease conferences, including ASM 2015 and ICAAC 2015.

“Taken together, our data suggest that Mul-1867 is a promising novel antimicrobial agent that has potent broad-spectrum antibacterial activity against clinically important microorganisms,” said Dr. Sushant Kumar, a member of the company’s advisory board. “TGV-Inhalonix has completed comprehensive preclinical and pilot clinical studies of Mul-1867 in Europe, showing good efficacy and safety profile in cystic fibrosis patients. Regarding development in the US, the company plans to conduct Investigational New Drug studies (IND) studies with an IND filing expected in 2018.”